申请人:PTC Therapeutics, Inc.
公开号:US20140235586A1
公开(公告)日:2014-08-21
The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.
本发明涉及通过给予本发明的化合物或组合物来治疗或预防与mRNA中的无意义突变相关的疾病的方法、化合物和组合物。更具体地,本发明涉及用于抑制与mRNA中的无意义突变相关的早期翻译终止的方法、化合物和组合物。